Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice
利用细胞启动子的慢病毒基因疗法治愈小鼠 1 型戈谢病
期刊:Molecular Therapy
影响因子:12.1
doi:10.1038/mt.2015.16
Maria Dahl, Alexander Doyle, Karin Olsson, Jan-Eric Månsson, André R A Marques, Mina Mirzaian, Johannes M Aerts, Mats Ehinger, Michael Rothe, Ute Modlich, Axel Schambach, Stefan Karlsson